STAT May 8, 2024
Two congenitally deaf children can hear for the first time after being treated with gene therapy, according to data presented at a conference Wednesday.
The results are “jaw-droppingly good, just shocking how good. It exceeded the wildest expectations of anybody who started this work,” said Larry Lustig, an otolaryngologist at Columbia University and an investigator on the study.
The new data come from a larger study by Regeneron Pharmaceuticals that will treat nearly two dozen children with a specific type of congenital hearing loss with gene therapy....